Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children
This is an exciting time for research and novel drug development in cystic fibrosis. However, rarely has the adage, “Children are not just little adults” been more relevant. This article is divided into two main sections. In the first, we explore why it is important to involve children in research....
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2022-07-01
|
| Series: | Children |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2227-9067/9/7/1080 |
| _version_ | 1797433770560716800 |
|---|---|
| author | Rebecca Dobra Siân Bentley Claire Edmondson Maxine Ovens Clare Saunders Christopher Short Gemma Wilson Jane C. Davies Andrew Bush |
| author_facet | Rebecca Dobra Siân Bentley Claire Edmondson Maxine Ovens Clare Saunders Christopher Short Gemma Wilson Jane C. Davies Andrew Bush |
| author_sort | Rebecca Dobra |
| collection | DOAJ |
| description | This is an exciting time for research and novel drug development in cystic fibrosis. However, rarely has the adage, “Children are not just little adults” been more relevant. This article is divided into two main sections. In the first, we explore why it is important to involve children in research. We discuss the potential benefits of understanding a disease and its treatment in children, and we highlight that children have the same legal and ethical right to evidence-based therapy as adults. Additionally, we discuss why extrapolation from adults may be inappropriate, for example, medication pharmacokinetics may be different in children, and there may be unpredictable adverse effects. In the second part, we discuss how to involve children and their families in research. We outline the importance and the complexities of selecting appropriate outcome measures, and we discuss the role co-design may have in improving the involvement of children. We highlight the importance of appropriate staffing and resourcing, and we outline some of the common challenges and possible solutions, including practical tips on obtaining consent/assent in children and adolescents. We conclude that it is unethical to simply rely on extrapolation from adult studies because research in young children is challenging and that research should be seen as a normal part of the paediatric therapeutic journey. |
| first_indexed | 2024-03-09T10:20:47Z |
| format | Article |
| id | doaj.art-e90c0ea83c6d4b048a66607d590776dc |
| institution | Directory Open Access Journal |
| issn | 2227-9067 |
| language | English |
| last_indexed | 2024-03-09T10:20:47Z |
| publishDate | 2022-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Children |
| spelling | doaj.art-e90c0ea83c6d4b048a66607d590776dc2023-12-01T22:01:57ZengMDPI AGChildren2227-90672022-07-0197108010.3390/children9071080Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include ChildrenRebecca Dobra0Siân Bentley1Claire Edmondson2Maxine Ovens3Clare Saunders4Christopher Short5Gemma Wilson6Jane C. Davies7Andrew Bush8National Heart and Lung Institute, Imperial College, London SW3 6LY, UKNational Heart and Lung Institute, Imperial College, London SW3 6LY, UKNational Heart and Lung Institute, Imperial College, London SW3 6LY, UKPlay Team, Royal Brompton Hospital, London SW3 6NP, UKNational Heart and Lung Institute, Imperial College, London SW3 6LY, UKNational Heart and Lung Institute, Imperial College, London SW3 6LY, UKPaediatric Respiratory Medicine Department, Royal Brompton Hospital, London SW3 6NP, UKNational Heart and Lung Institute, Imperial College, London SW3 6LY, UKPaediatric Respiratory Medicine Department, Royal Brompton Hospital, London SW3 6NP, UKThis is an exciting time for research and novel drug development in cystic fibrosis. However, rarely has the adage, “Children are not just little adults” been more relevant. This article is divided into two main sections. In the first, we explore why it is important to involve children in research. We discuss the potential benefits of understanding a disease and its treatment in children, and we highlight that children have the same legal and ethical right to evidence-based therapy as adults. Additionally, we discuss why extrapolation from adults may be inappropriate, for example, medication pharmacokinetics may be different in children, and there may be unpredictable adverse effects. In the second part, we discuss how to involve children and their families in research. We outline the importance and the complexities of selecting appropriate outcome measures, and we discuss the role co-design may have in improving the involvement of children. We highlight the importance of appropriate staffing and resourcing, and we outline some of the common challenges and possible solutions, including practical tips on obtaining consent/assent in children and adolescents. We conclude that it is unethical to simply rely on extrapolation from adult studies because research in young children is challenging and that research should be seen as a normal part of the paediatric therapeutic journey.https://www.mdpi.com/2227-9067/9/7/1080cystic fibrosischildrenclinical trialsethicspatient-centred trials |
| spellingShingle | Rebecca Dobra Siân Bentley Claire Edmondson Maxine Ovens Clare Saunders Christopher Short Gemma Wilson Jane C. Davies Andrew Bush Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children Children cystic fibrosis children clinical trials ethics patient-centred trials |
| title | Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children |
| title_full | Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children |
| title_fullStr | Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children |
| title_full_unstemmed | Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children |
| title_short | Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children |
| title_sort | going the extra mile why clinical research in cystic fibrosis must include children |
| topic | cystic fibrosis children clinical trials ethics patient-centred trials |
| url | https://www.mdpi.com/2227-9067/9/7/1080 |
| work_keys_str_mv | AT rebeccadobra goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT sianbentley goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT claireedmondson goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT maxineovens goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT claresaunders goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT christophershort goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT gemmawilson goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT janecdavies goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren AT andrewbush goingtheextramilewhyclinicalresearchincysticfibrosismustincludechildren |